Accessing Unapproved Therapeutic Products

There are a number of ways that patients can gain access to products that have not been approved for use in Australia.

Special Access Scheme

The Special Access Scheme (SAS) refers to arrangements which provide for the import and/or supply of an unapproved therapeutic good for a single patient, on a case by case basis. Applications for special access are coordinated by the Office for Research. Contact ResearchGovernance@mh.org.au.

Clinical Trials

There are two schemes under which clinical trials involving therapeutic goods may be conducted in Australia – CTN and CTX schemes. Further information, including copies of any forms that need to be completed, is available on the TGA website.

Authorised Prescribers

TheTGAis able to grant medical practitioners authority to prescribe a specified unapproved therapeutic good or class of unapproved therapeutic goods to specified recipients or classes of recipients. Under theAuthorisedPrescriber Scheme (APS), individual medical practitioners can apply to be granted authority to become an “AuthorisedPrescriber” of a specified unapproved therapeutic good (or class of unapproved therapeutic goods) to specific groups of patients with a particular medical condition.

Therapeutic goods not included on the Australian Register of Therapeutic Goods (ARTG) cannot legally be supplied unless the TGA grants an exemption. The Authorised Prescriber Scheme allows medical practitioners who become Authorised Prescribers to access and legally supply an unapproved therapeutic good or class of goods to appropriate patients/categories of patients. Only registered medical practitioners are able to become Authorised Prescribers.

To become an Authorised Prescriber, a medical practitioner must first obtain approval from a human research ethics committee (HREC) or endorsement from a specialist college. They will then need to submit an application to the TGA for evaluation. This submission should include:

a completed Authorised Prescriber Scheme application form

evidence of either approval from a HREC or endorsement from a specialist college.

Following consideration of an Authorised Prescriber application, the HREC will issue (if application is approved) a formal letter of approval. This letter should be provided to the TGA together with the completed Authorised Prescriber Application Form – ensure the indication listed on the application form is the indication approved by the HREC.

1. Explanatory cover letter to the HREC

Submit a cover letter which includes the following information:

the reason for the Authorised Prescriber application; and

the name of each registered medical practitioner seeking endorsement to become an Authorised Prescriber.

Provide details of exactly how the drug or device will be obtained/supplied.

Will an agreement between Melbourne Health and the provider be required? If so, provide an electronic copy of the agreement from the supplier for review and execution by the Office for Research.

Authorised Prescribers are responsible for obtaining the unapproved therapeutic good. If the Authorised Prescriber is an employee of Melbourne Health, these arrangements will be made by Melbourne Health on behalf of the approved Authorised Prescriber.

3. Submit CV's

Submit a curriculum vitae (CV) for each registered medical practitioner.

The clinical justification for use of the unapproved good should provide sufficient evidence to demonstrate that this use is appropriate, considering the availability of any approved goods that may be suitable alternatives. Specifically, provide information on:

the indication for which the good will be used

the seriousness of the condition

the expected benefits of the proposed treatment versus its potential risks

It should also address the circumstances where there are approved treatments for the same indication, specifically:

have they been attempted?

will they be attempted prior to supplying the unapproved good?

why are they inappropriate?

why is the proposed unapproved good a more appropriate option than any approved available alternative

how the risk associated with the use of an unapproved good will be managed

the monitoring that will be undertaken

the process for investigating and reporting adverse events

6. Product information or investigator brochure

Provide product information or an investigator brochure including toxicology and safety information.

7. Informed consent

Provide a copy of the template of the informed consent document to be provided to each patient receiving the unapproved therapeutic product. Often the template will be provided by the supplier of the unapproved therapeutic product. The Authorised Prescriber must obtain the written informed consent of each patient for whom they prescribe the unapproved product. This consent form must be in plain and simple English and include statements about the following:

that the TGA has not evaluated the unapproved good’s safety, quality and efficacy

that the product is not approved for marketing in Australia;

the possible benefits of treatment and any risks and side effects that are known;

the possibility of unknown risks and side effects; and

any alternative treatments using approved products which are available.